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ClinicalTrials.gov public records Last synced Oct 17, 2026, 4:05 PM EDT

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Showing 1105–1128 of 1,710 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional Results available

Stem Cell Transplantation for Hurler

NCT00176917
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting No phase listed Observational

Human Epilepsy Genetics--Neuronal Migration Disorders Study

NCT00041600
View directory record Official record
Conditions
Brain Malformation, Neuronal Migration Disorder, Cognition Disorder, Epilepsy
Interventions
Not listed
Lead sponsor
Harvard University Faculty of Medicine
Other
Eligibility
Not listed
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 17, 2026, 4:05 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Wearable Gait Sensors

NCT06628934
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
18 Years to 75 Years
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting Phase 1Phase 2 Interventional

TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)

NCT07190300
View directory record Official record
Conditions
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
Interventions
Tulmimetostat, Darolutamide, Abiraterone, Prednisone/Prednisolone, Androgen Deprivation Therapy (ADT)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
181 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
8
States / cities
Birmingham, Alabama • Iowa City, Iowa • Westwood, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Tissue Sample Study for Mitochondrial Disorders

NCT01803906
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
6,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Amyloid Deposition in Adults With Down Syndrome

NCT01303133
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
30 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2018
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration

NCT00878436
View directory record Official record
Conditions
Prostate Cancer, Prostatic Neoplasms
Interventions
Panobinostat, Bicalutamide
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
4
States / cities
New Brunswick, New Jersey • Lake Success, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2018 · Synced Oct 17, 2026, 4:05 PM EDT
Active, not recruiting Phase 1 Interventional

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT06054893
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 4 Interventional Results available

Comparison of Keppra and Clonidine in the Treatment of Tics

NCT00370838
View directory record Official record
Conditions
Tic Disorders, Tourette Syndrome
Interventions
Levetiracetam, Clonidine
Drug
Lead sponsor
Harvey S. Singer
Other
Eligibility
7 Years to 19 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 7, 2011 · Synced Oct 17, 2026, 4:05 PM EDT
Terminated Phase 2 Interventional Results available

A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease

NCT02494778
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine
Drug
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
11
States / cities
Los Angeles, California • Englewood, Colorado • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2021 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 1 Interventional

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

NCT05573698
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 29, 2024 · Synced Oct 17, 2026, 4:05 PM EDT
Completed No phase listed Observational

A Natural History Study of Canavan Disease

NCT02851563
View directory record Official record
Conditions
Canavan Disease
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
Up to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting Phase 2 Interventional

Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

NCT07766980
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
Interventions
VM100 Nutritional Supplement
Drug
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Completed No phase listed Observational

Tourette Discrimination (TD) Stigma Scale

NCT05696769
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tic Disorder, Childhood, Tics
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
8 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 17, 2026, 4:05 PM EDT
Terminated Phase 1 Interventional

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

NCT00215527
View directory record Official record
Conditions
Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 21, 2013 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 3 Interventional Results available

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

NCT04966741
View directory record Official record
Conditions
Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 5 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Aurora, Colorado • New York, New York • Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 17, 2026, 4:05 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569
View directory record Official record
Conditions
GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2
Drug
Lead sponsor
Terence Flotte
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 17, 2026, 4:05 PM EDT
Completed Phase 2 Interventional Results available

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

NCT03406962
View directory record Official record
Conditions
Inherited Metabolic Disorders (IMD)
Interventions
MGTA-456
Drug
Lead sponsor
Magenta Therapeutics, Inc.
Industry
Eligibility
0 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
2
States / cities
Minneapolis, Minnesota • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies

NCT03059420
View directory record Official record
Conditions
Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome, Brown Syndrome, Marcus Gunn Syndrome, Strabismus Congenital, Horizontal Gaze Palsy, Horizontal Gaze Palsy With Progressive Scoliosis, Facial Palsy, Facial Paresis, Hereditary, Congenital, Third Nerve Palsy, Fourth Nerve Palsy, Sixth Nerve Palsy, Synkinesis, Ocular Motility Disorders, Levator-Medial Rectus Synkinesis, Athabaskan Brainstem Dysgenesis, Tongue Paralysis, Ninth Nerve Disorder, Fifth Nerve Palsy, Seventh Nerve Palsy, Eleventh Nerve Disorder, Twelfth Nerve Disorder, Vagus Nerve Paralysis, Moebius Sequence
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Day and older
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

NCT05462106
View directory record Official record
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 17, 2026, 4:05 PM EDT
Recruiting No phase listed Observational

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

NCT05843552
View directory record Official record
Conditions
Gaucher Disease
Interventions
no intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 17, 2026, 4:05 PM EDT